International Journal of Technology Assessment in Health Care最新文献

The National Institute for Health and Care Excellence (NICE) early value assessment (EVA) was launched in 2022 as a process to assess new technologies that have the potential to meet an unmet need or demand. The recommendations that result from the process are best viewed as a type of managed entry agreement - that is, time-limited and conditional on further evidence being generated. This commentary, from authors in PenTAG (an external assessment group involved in assessing medical technologies for NICE, based at the University of Exeter), explores the challenges that have arisen during 3 years of performing EVAs, offers some thoughts on EVA's role in evidence generation, and their fit in NICE's wider evidence landscape. The commentary identifies areas for potential improvement in terms of timelines, scoping and protocol development, searching, reviewing, and economic modeling. Many of the suggested changes are relatively minor tweaks to the process, or requests for clearer guidance or expectation management. We conclude that, with some changes to the EVA process and its accompanying guidance, the assessments could become more efficient. In summary, the EVA represents NICE's life cycle approach in their HealthTech program, wherein evidence is collected along the life cycle to help monitor initial assumptions and recommendations made. The process is designed to continuously capture incremental innovation over the lifetime of a medical device. As such, EVAs reflect a small but important shift in how health technology assessment is practiced.

Objectives: The reimbursement of, and subsequent patient access to, high-risk medical devices (MD) and in vitro diagnostics (IVD) across Europe often vary. The Health Technology Assessment Regulation (HTAR) aims to standardize clinical evaluations through Joint Clinical Assessments. Still, national differences in reimbursement frameworks and evidence integration for MD/IVD may impede the realization of HTAR's expected benefits. This review aims to map existing reimbursement frameworks for high-risk MD/IVD, identify key oversight structures, and evaluate the use of comparative effectiveness and safety evidence in reimbursement decisions across the EU/EEA/UK.

Methods: A scoping review was conducted according to the registered protocol (osf.io/65bdk) and was reported following the PRISMA-ScR guidelines. Results were validated through direct engagement with national organizations.

Results: Reimbursement frameworks across the EU/EEA/UK for MD/IVD vary significantly. Of the thirty-four countries reviewed, twenty-three incorporate HTA for MD/IVD reimbursement decisions; of these, only eleven countries have a formal HTA process as part of reimbursement pathways. Eight countries have structured mechanisms to address safety and effectiveness evidence uncertainty. Furthermore, twelve countries have primarily centralized processes, while six rely on regional or local decision-making.

Conclusions: This review highlights the variations in how countries integrate HTA into reimbursement frameworks for MD/IVD, how the national decisions are implemented, and how the evidence uncertainty is assessed. Some countries have a well-established reimbursement framework with formal HTA components, whereas others rely on ad hoc HTA processes. Understanding these differences can help optimize the use of HTAR-generated evidence. Further research is needed to capture ongoing reforms in response to the HTAR.

Objectives: This scoping review aims to synthesize the literature on pediatric health technology assessments (HTAs) and map out the challenges of assessing new technologies for use in children, with a particular focus on pharmaceutical interventions.

Methods: Conducted in accordance with the Joanna Briggs Institute Methodology, this scoping review addressed HTAs in the pediatric domain through searches of PubMed, Embase, Web of Science Core Collection, and EconLit through 22 January 2024, as well as the gray literature. Sources were excluded if they (i) were a clinical trial investigating a specific technology or an HTA of that technology, (ii) did not address the challenges of HTAs, or (iii) had no relevance to pediatrics. Two authors performed screening and data extraction independently and in duplicate.

Results: One hundred and three reports were included. Of these, sixty were full journal articles, twenty-three were conference abstracts, and twenty were guidelines, reports, and other documents. Two important themes emerged from this work. The first was the unique position of children within society and the resulting difficulty of incorporating them within a population-wide HTA system. The second was the uncertainty that characterized pediatric HTAs due to data constraints and either a lack of guidance by HTA bodies or variations in guidance between bodies.

Conclusions: Many factors inherent to children, including the heterogeneity of pediatric disease populations, long-term outcomes, and children's distinct social positions, render conducting pediatric HTAs challenging. Innovative approaches are required to address these challenges and respond to the needs of pediatric populations.

Objectives: The introduction of a new healthcare technology within the technological facilities of a hospital is a complex action that must go through the mandatory decision-making process of health technology assessment (HTA). Nowadays, developing a universal HTA model poses a significant challenge within the current landscape. This paper describes the proposal of a novel supporting healthcare technology evaluation toolbox, aligned with the principles of the European Network for Health Technology Assessment (EUnetHTA) shared by the Regulation (EU) 2021/2282 on Health Technology Assessment (HTAR).

Methods: The proposed toolbox relies on a MATLAB-based multicriteria algorithm that mirrors the evaluative procedure following the hierarchical framework of the analytic hierarchy process. The evaluation framework involves clinical and non-clinical aspects leading to the choice of the best alternative, among the evaluated technologies, to be introduced in the technological infrastructure of the hospital. Moreover, the toolbox incorporates robust economic analysis capabilities, crucial for determining the requisite number of annual hospital procedures to ensure economic equilibrium and mitigate financial risks. Additionally, it computes the payback period, essential for evaluating the economic feasibility of technology investments. HTA evaluations at San Giovanni Addolorata Hospital demonstrate its application.

Results: The toolbox exemplifies its efficacy in supporting informed decision-making processes, regarding the adoption of technologies like robotic systems for neurosurgery and angiographic systems, in terms of economic sustainability and clinical effectiveness.

Conclusions: This study underscores the toolbox's role in advancing HTA methodologies and enhancing the efficiency and sustainability of healthcare technology integration.

Objectives: Under the Universal Health Care Act of the Philippines, all health technologies should undergo health technology assessment. This manuscript details the process of the development of the Philippine guidance document for the use of real-world evidence (RWE) in the clinical evaluation of health technologies.

Methods: This study consisted of two phases. Phase 1 was a comprehensive, systematic review of all available HTA methods guides and literature related to the use of RWE in the clinical evaluation of health technologies. Based on the results of the review, a draft HTA methods guide on the use of RWE was created. Phase 2 was a validation study by expert consultation through key informant interviews (KIIs), and pilot assessment of the methods guide.

Results: Seventy-nine articles and nine guidance papers were included, with pertinent information extracted and organized into sections. The first draft covered definitions of RWE, guidance for RWE utilization, scoping and selecting RWE, critical appraisal, data extraction, and synthesis and analysis of RWE. Changes were made to this draft based on the KIIs and pilot assessment results to produce the final output of the methods guide.

Conclusions: This document describes the process of creating a Philippine guidance document that covers the definition of RWE and the appropriate methods for conducting systematic search, screening, critical appraisal, data extraction, data analysis, and synthesis of RWE.

Background: Developing therapies for rare diseases is challenging due to limited evidence and high degrees of uncertainty regarding the value of new treatments. Clinical expert judgment can inform modeling assumptions and address areas of uncertainty in reimbursement submissions. As current protocols do not adequately address the challenges faced in rare diseases, this research aimed to generate recommendations for the collection and reporting of clinical expert judgment in rare diseases.

Methods: An international group of industry, payer, and patient experts with a background in rare diseases participated in a roundtable meeting, which aimed to identify practical challenges in and solutions for gathering clinical insights to aid reimbursement decisions for rare disease therapies. Recommendations were cocreated through iterative discussions and group agreement.

Results: Developers should proactively identify uncertainties that expert judgment can address, in parallel with early evidence generation planning. Expert judgment method(s) depend on the uncertainties, with those key to decision-making requiring more robust and time-intensive methods. For highly complex and uncertain topics, methods should facilitate consensus building and expression of diverse views. Given the scarcity of rare disease experts, a high time burden falls on a few experts. Developers should engage diverse stakeholder groups to integrate broader clinical perspectives and reduce reliance on specific individuals while approaching conflicts of interest pragmatically and transparently.

Conclusions: These recommendations create a blueprint for developers of rare disease therapies to conduct high-quality clinical expert judgment studies. Hence, developers can present more robust evidence to inform key areas of uncertainty in reimbursement decisions, where empirical evidence is unavailable.

Aims: Health technology assessment (HTA) for medical devices (MDs) is essential for adoption decisions, but the sector's particularities studied here defy regulatory frameworks. In Brazil, the National Policy for Health Technology Management (PNGTS) provides guidelines for HTA, but the reimbursement of MDs in the Brazilian National Health System (SUS) still faces challenges. This study aimed to identify and validate relevant domains and attributes for HTA of MDs in the SUS, considering the perspectives of various stakeholders.

Objectives: To analyze and validate the essential domains and attributes for conducting HTA studies focused on the reimbursement of MDs in the SUS.

Methods: A baseline systematic review was performed, which was followed by two additional stages: a survey with 115 participants and a Delphi panel with 33 experts. Likert scales were used to assess the importance of the domains and attributes, along with open questions to collect suggestions and comments.

Results: The domains "clinical benefits," "evidence ecosystem," and "budget impact" were considered fundamental. "Social participation" showed high variability in response, indicating the need for greater engagement and clarity in participation mechanisms. The inclusion of the "public policy" domain emphasizes the importance of aligning government policies with population needs.

Conclusions: This study reinforced the relevance of a multidisciplinary and participatory approach in HTA for MDs, with a focus on clinical outcomes, real-world evidence, and continuous monitoring. Overcoming the identified challenges, such as information gaps and the need for robust methods, is crucial for improving the reimbursement of MDs in the SUS.

Background: The Dutch National Health Care Institute (ZIN) advises the Dutch Minister of Health on the basic benefit package using Health Technology Assessment (HTA), focusing on necessity, clinical effectiveness, cost-effectiveness, and feasibility. Despite the huge environmental impact of the healthcare sector, this impact is not taken into consideration. Several methodological approaches to integrate the environmental impact into HTA have been proposed, including information conduit, integrated evaluation, parallel evaluation, and environment-focused evaluation. There is significant uncertainty as to which approach is the most appropriate. Therefore, it is important to understand stakeholders' perspectives on these approaches.

Objectives: To explore Dutch stakeholders' perspectives on integrating environmental impacts into HTA and assess preferred methods and challenges.

Methods: A qualitative study using a focus group with members from ZIN (n = 7) and individual interviews (n = 7) with experts in HTA, market access, and reimbursement. Interviews were transcribed and analyzed thematically.

Results: Stakeholders highlighted the importance of addressing environmental impacts such as pharmaceutical pollution, greenhouse gas emissions, and waste. Integrated and parallel evaluations were preferred, but barriers such as data gaps, methodological complexity, and lack of guidelines were noted.

Conclusion: Incorporating environmental impacts into HTA is crucial but requires clear guidelines, better data, and stakeholder collaboration to support sustainable healthcare practices.